No. 41 "Making PPI More Accessible: Patient and Citizen Involvement in Medical Care That Can Be Started Tomorrow

Seminar Date : March 5, 2024 (Tuesday)

Video distribution period : September 30, 2024 (Mon.) - September 30, 2025 (Tue.) [1 year

Opening remarks

In recent years, various related parties and organizations have been engaged in activities to promote Patient and Public Involvement (PPI) in healthcare, and recently it has been recognized that patient participation is necessary not only in the field of research but also in all aspects of healthcare policy. We hope that this event will provide an opportunity for you to think about PPI together and take a step forward.

Lecture

Subject 1: The Latest Trend of PPI: Toward Further Promotion of PPI
Ms. Akiko Yoshida, Senior Researcher, Pharmaceutical and Industrial Policy Research Institute

PPI is a concept first introduced in the UK. The term "patient-citizen engagement" is often translated as "patient-citizen participation" in Japanese, and it was originally defined in the research field as research conducted with or by patients/citizens. The concept has evolved into the idea that patients are not only the beneficiaries of established medical care, but also partners in the creation of good medical care.
For pharmaceutical companies, PPI is becoming a fundamental principle to achieve the best possible outcome for patients and their families. As to why PPI is necessary for pharmaceutical companies, it is because we have realized that there is a gap between the ideas and needs of pharmaceutical companies and patients and their families, and we believe that there is a common understanding in the pharmaceutical industry that we cannot create drugs that are valuable to patients unless we understand the true needs of patients at an early stage. We believe that this is a common understanding in the pharmaceutical industry.

According to the survey results, more than 90% of patients answered that PPI activities are meaningful. However, only about 60% are aware of PPI activities and only 10% participate in PPI activities, and further recognition and participation are expected in the future.

PPI is a rapidly growing and increasingly important field in the creation of pharmaceuticals worldwide, and in other countries, the government has established systems to listen to patients' voices and promote patient participation. We hope to see such a system in Japan as well. Although PPI is not yet well recognized by patients in Japan, the number of PPI initiatives and opportunities for participation is increasing, not only in the pharmaceutical industry, but also in patient groups and academic societies, and I think it is becoming easier and easier to participate. I hope that you will actively participate in these activities.
In the pharmaceutical industry, we would like to understand the true needs of patients and their families, create drugs that are of value to patients and their families earlier, and work toward the realization of a better medical environment.

Subject 2: Expectations for "Participation"-People's Thoughts
Matsuyo Kamada, Representative Director, Association of Families with Dementia

I joined the Family Association in 1990 to learn more about dementia, and I have served as President since 2023. I was originally a nurse working at a university hospital, but my father-in-law suffered a cerebral hemorrhage and became paralyzed on the left side of his body, resulting in higher brain dysfunction. At that time, I realized how difficult it was to care for him at home, and after raising my children, I worked in the field of home nursing, day care services and special care.
In 2004, my father was diagnosed with Alzheimer's disease at my parents' home in Saga, and four years later, my mother fell ill with the same disease. Along with my father's diagnosis, I went to long-distance care every month for 11 years. During that time, my mother-in-law in Kyoto was also diagnosed with Alzheimer's disease and was cared for at home, and I am both a family caregiver and a professional.

The Family Association was formed in January 1980 and is now in its 44th year as a public service corporation. We have 9,400 members, mainly individuals and their families, but also anyone who is interested in dementia can join.

The most important activity of the Family Caregiver's Association is the meeting. We hold meetings where caregivers present their caregiving situations to each other, and where they can exchange information about their own caregiving situations and receive information from doctors and other professionals to make their caregiving as easy as possible and to create a comfortable environment for the patient. We continue to hold these meetings even now, 44 years later. In the bulletin of those days, we reported that we met people who were taking care of similar patients and learned that there were others who were having a more difficult time than we were, which made us feel better and gave us courage. Even today, there is an overflow of information about dementia and care services, but the pain of having a family member with dementia can only be understood by those who have experienced the same thing. In addition, we also publish newsletters, provide telephone counseling, and raise awareness about dementia.

As part of our activities, we also submit requests to the Minister of Health, Labor and Welfare. I think this is the starting point of PPI. We have written more than 70 requests to the government. After submitting these requests, we meet with representatives of the government and officials of the Ministry of Health, Labor and Welfare to discuss our actual situation. However, it has always been a one-way process, in which we simply said that we had accepted the request.

On January 1 of this year, the Basic Law on Dementia came into effect to promote the realization of a symbiotic society. Paragraph 3 of Article 3 of the law states, "Ensure opportunities for people with dementia to express their opinions on matters directly related to them and to participate in activities in all areas of society. I think it can be tied equally with the expectations for participation and PPI. I have high expectations for the creation of a place where companies and government agencies can interact with the activities of the Family Association to deliver the voices of the people involved, rather than the one-way "we have heard from you" situation that has been the norm up to now.

However, I also have some concerns about participation. First of all, I have a sense of resistance to PPI, which is written in Roman characters. I feel as if I am being asked to do something difficult. Also, I am concerned that I don't know what participation means or what I should do. I don't know what the difference is between participation and involvement, and I have anxiety about being asked to participate in something I don't understand. There is also a concern that even if there is a preliminary explanation, it may not be enough for them to understand the pathology or the mechanism of disease development.

We have very high expectations for participation. We believe that participation can lead to a better life for the people involved and for future people involved. We understand that the participants need to learn, but they cannot participate without understanding the terminology and pathology, so we expect support from experts and companies. I am looking forward to "Togetherness", where participation is improved by combining what we learn with our own experiences, and where we can continue to evolve as we think with the people involved and create drugs. Through participation, we hope that the experiences of those involved will lead to contributions to society and a sense of fulfillment in life, and that through dialogue, mutual understanding and synergy will be created among those involved, thereby improving the lives of patients and their families.

Subject 3: Hopes of a Small Patients' Association, Thoughts on PPI
Ms. Ayako Hiroishi, MLC Patients' Association

I usually work as an office worker and run the patient group mainly with my husband. We have a first grade daughter and a 5 year old son, and we started the patient group 3 years ago when my son was diagnosed with MLC, a macrocephalic leukoencephalopathy with subcortical cysts, when he was 2 years old. He is happy and healthy and attends preschool, although he wears a head guard because the disease causes him to have a large, wobbly head and trauma to the head can accelerate the progression of the disease. He is a very normal 5-year-old boy who laughs a lot.

One of the main characteristics of MLC is macrocephaly, a large head. Developmental delay, incoming intellectual disability, epilepsy, etc. It is said to be a condition that makes a teenager wheelchair bound, but the symptoms and speed of progression vary from person to person. My son was developmentally delayed and attended a rehabilitation center, but when he finally started walking at the age of two, his doctor told him that he had a disease that would make him a wheelchair user in his teens. Even so, it was a hellish time when I had to look forward.

MLC is one of the government-designated intractable diseases, progressive leukoencephalopathy, which is also designated as a chronic pediatric disease. The advisor of the patients' association is Dr. Toshiyuki Yamamoto of Tokyo Women's Medical University. It is said that there are currently about 20 people diagnosed with MLC in Japan.

Four years ago, the University of Barcelona published a paper on gene therapy for MLC, which was the starting point for the activities of the Patients' Association. This paper gave us hope that maybe there is a cure for the disease, but it is just not known and researched at the moment, and we wanted to make the disease known to as many researchers as possible anyway.

Here are some of the challenges we have experienced so far on the patient side.
We were in the pattern of not having a patient group when we were diagnosed. We contacted representatives of other rare disease patient associations that had already been established and were willing to share their stories with us, and then we were introduced to them, gradually expanding our circle of activities. One of the patient groups introduced me to Dr. Nishikawa of the NPO AASJ, who was going to hold an MLC study session, and Dr. Yamamoto of the Tokyo Women's Medical University, who was an advisor to the patients' group, also attended. At the time of my diagnosis, it would have been nice to have an organization that could gather information together with me. However, we were still gathering information in a tense situation, as if we had not yet accepted the reality of the situation, so it would have been meaningless unless they were as excited and desperate to gather information as we were. We are still gathering information.
I would like to receive advice from the pharmaceutical companies on the challenges of setting up a patient association, such as what information should be collected with an eye to future clinical trials, PPI activities, etc., as well as what information should be accepted as a patient association if it is complete.
We would also like to have a mechanism to match disease research with researchers.

The first issue that I am currently aware of is the patient registry. I think the type of registry will be different for each disease, but I feel that this is exactly the kind of registry that requires cooperation among researchers, doctors, and pharmaceutical companies. I have also heard that data held by patient associations is highly flexible and easy to use. I also think that as a patient association, we need to learn what steps we need to take before clinical trials.
The second issue is awareness-raising. I also feel that we need to raise awareness not only about MLC, but also about white matter dystrophy and rare diseases similar to MLC, respectively. We want to do something for the mothers who have to provide medical care every few hours. I also wonder how to fund research in Japan, although there are large foundations in Europe and the United States.

Furthermore, I believe that the number of patients with rare diseases will continue to increase as scientific and medical technologies advance and things that were previously unknown become clearer. I hope that I can create a stir in the PPI as a member of a small patients' association so that such patients will not be left in despair in this country. In Mr. Yoshida's lecture, he mentioned that encounters will expand through PPI, and I agree. I really don't think it's all bad.

Subject 4: How will you tackle PPI?
～How do you tackle PPI?
Ms. Miho Goto, Representative Director, NPO CNS Network Council

I run a patient group and family association related to the psychiatric and neurological fields at the CNS (Central Nervous System) Network Council.
As for my own background in running a family and patient group specializing in psychiatry, when I was a teenager, I lost a close relative of the same generation to a mental illness. I was shocked at the fact that I could not recognize mental illness even though I was closest to it, and I had a hazy feeling inside myself, so I went to university to I was shocked by the fact that I was unable to recognize mental illness even though I was closest to it.
　In the nursing department, I was involved in the activities of family associations, and my graduation thesis was also related to family associations. After working as a nurse at a psychiatric hospital, I began working as a clinical trial coordinator and clinical trial coordinator at a medical institution that develops drugs in 2007. The activities of the party, patient, and family associations that I had continued as a volunteer were organized into an organization called the CNS Network Council in 2014. Currently, I am a board member of the Institute for the Foundation of Medical Development Institutions, a trustee of the Clinical Trial Support Foundation, and the chairmanship of the Japan SMO Association (which supports the implementation of clinical trials at medical institutions).
　What is PPI? And what does patient/citizen engagement actually do? I would like to talk about PPI in the context of our usual activities.
Patient/citizen involvement does not mean that we have to suddenly make a big move, but first of all, I would like to tell people that we should speak out about what we are doing, not only within our own organization, but also outside our organization. One of the things we could do is to try to reach out to the government or pharmaceutical companies as an organization. For example, I think it is quite major where consumer viewpoints and opinions are incorporated in general products and services, but what about in the development of drugs? I think it would be a good idea to give our opinions a little more input.
　In working on the PPI, I think it would be good to learn first, and then transmit our voice.
　First of all, we need to learn how to manage patient groups and other organizations as tips for approaching the government and pharmaceutical companies as an organization, and how to develop and continue the organization as a sustainable one, rather than learning specifically about PPI. I think it is necessary to learn how to develop and sustain an organization as a sustainable one. As a form of literacy, I think it is important to learn about health and medical care with regard to diseases that we and our families have. In a world where we can easily obtain a variety of information on the Internet, I feel that it is necessary to develop the ability to discern which information is true and beneficial to us.

After learning, I believe it is important to send out our voices and share our thoughts and opinions so that we can communicate our ideas and opinions. This means sharing concerns and anxieties with other parties/families, discussing about medicines, telling what you want from the government, and delivering your wishes to pharmaceutical companies about what you wish they had or your thoughts about clinical trials. I also think we need to consider whether holding online meetings is really good for the parties/families.

We often talk about how to tackle PPI and how it would be good to have an open dialogue with both parties and their families in a relationship where it is OK for me and OK for you. I tell them that it is very important to express themselves honestly, to listen to others, to express themselves and others with care, and to communicate with each other.
When you hear about PPI, you may wonder where to start and how to tackle it. I think it is important not to think too hard, but to start with what you can do, and to take action.

Panel Discussion

Ikezaki (titles below: titles omitted)

I myself am a patient with a neurological intractable disease, and I am currently the secretariat of PPeCC, which has been active in the field of intractable diseases.
I would like to ask you all to talk about making PPI more accessible to the public. Now, I would like to ask you to be more specific. Mr. Yoshida talked about the current situation in Japan and examples from other countries. What do you think is needed to involve the public more in PPI?

Yoshida

As a pharmaceutical company employee, I myself am a citizen when I go home. I think it is important for everyone to be interested in and participate in various opportunities. Then you can talk to your family and friends about what you feel, and when they start to participate, the circle will expand. Today, I have heard many words that have a broadening effect, such as understanding, cooperation, and encounters between both parties, and I believe that this is what PPI is all about. I hope that this will lead to the expansion of the circle of people.

Ikezaki

It may sound ostentatious to talk about dialogue and collaboration, but I thought that telling people close to you about diseases and medicines is also a PPI. Mr. Kamata, you mentioned that your organization has already been implementing policy proposals, etc., and that you have expectations and concerns about participating in the development of medicines.

Kamata

I was wondering if it was okay for patients to participate in the drug scene. I had the feeling that it was not a good idea for patients to participate in the drug scene. I have been approached by doctors about clinical trials, but I still feel that I have not received other information. I think it is necessary to obtain information on one's own, but the patients and their families, who are living with dementia to the fullest, are not able to obtain such information. I think it is necessary to start by shortening the distance between patients and their families by delivering the information to places where they can easily look at it.

Ikezaki

I think it is very difficult for patients to get information by themselves while living with the disease. I think that the closer you can get information, the closer you can get to PPI. Mr. Hiroishi, thank you very much for sharing your own experience and how you are paving the way for the future. You mentioned that you have been in contact with various senior organizations, and you also mentioned that matching with researchers may be necessary in the future.

Hiroishi

If there is a doctor who is prepared to help patients with diseases and their families, I think that alone would be a great help to the families. I am sure that even if such an existence were to emerge, the patients would be cheered up. I know it is really difficult, but my hope is that some kind of policy, organization, or function would be established in this area.

Ikezaki

I think that matching is important for the hope that life after tomorrow may change. Mr. Goto has a lot of knowledge and experience within his organization, and I think that his message of disseminating this knowledge and experience outside of the organization, even if it is not necessarily large, is something that all of the participants today could feel close to home. I think that the issues that will come up in the course of promoting PPI in the future, such as what is necessary for a flat dialogue, would be interesting to hear your opinions on this topic.

Goto

I am running a group of people concerned and a family group, and in order to be able to talk with medical personnel and other concerned parties on an equal footing, I tell them that they should first learn about their disease, learn about the medications they are taking, and be able to accurately convey their symptoms to the doctor within the limited time available at the doctor's office. I actually try to compile this information in my notebook. I believe that we can gain information, understand, and learn from the opinions of various people by participating in patient and family meetings.

Ikezaki

The subtitle of today's session, "Patient and Citizen Involvement in Medical Care," suggests that it may be difficult to immediately start something tomorrow, but many of you may be hoping to take home some hints on what you can do to put things into practice. I would like to hear your opinions on what you would like to do and what you should try to do, from the viewpoint of what you can actually do and how you can put it into practice.

Yoshida

From the standpoint of the pharmaceutical industry, I think these opportunities are very important. I think it is important to talk about your experiences with PPI, including my own, and to communicate them to the outside world. I think it is also important to listen to your experiences.

Ikezaki

As Mr. Hiroishi mentioned, I think it will lead to the implementation of PPI if each one of us transmits information from our own standpoints through our own experiences. How about you, Mr. Kamada?

Kamata

First of all, since we are an association of people concerned, we are able to inform people and their families that such a place is available for them and that such an era is coming. The second is that the first touch is from the doctors and pharmacies, so if the people in those places can tell us and our families that such a place is available, and if the information comes from the closest place to us, we will be able to understand that, yes, times have changed, and that medicine is not made by specialists, but by patients together. I thought it would be nice if we could learn something about the fact that it is not the specialists who make the medicines, but the patients who make the medicines together. For that purpose, I thought that doctors, pharmaceutical companies, and we should tell our members and other people that such a place is available and that the times have changed.

Ikezaki

Mr. Hiroishi, what do you think?

Hiroishi

I would like to have my thoughts heard by the people around me, such as large organizations and pharmaceutical companies, but I think it is important to let the people around me know and to talk to the people I can talk to. When I sent out my thoughts on social media, there were many people who reached out to me. I think it is also very important to meet people.

Ikezaki

This is something that can be practiced today, not tomorrow. I would like to ask you, Mr. Goto, if you have any ideas on what we can do from tomorrow.

Goto

I have been talking about learning and communicating, and I think it is important to communicate. If we don't communicate what we are doing on SNS or any other means, people won't know what we are doing. In addition, I think it would be good if the pharmaceutical companies would also disseminate information on good practices so that both parties can catch up with each other.

Ikezaki

It would be great if not only patient groups but also pharmaceutical companies and various stakeholders could share their experiences and knowledge. I am sure that everyone's approach to PPI is different, but I hope that today's lectures and panel discussion will make you feel a little more familiar with PPI and encourage you to take some action. I would be very happy if today's lecture and panel discussion could make you feel a little more familiar with PPI and make you want to try to work on it.

Provision of information

Introduction of the "Association for Creating a Society with Easy Access to Clinical Trials for All

The "Society for Creating a Society Where Everyone Has Easy Access to Clinical Trials" (hereafter referred to as "the Society"), which was launched in June 2023, was introduced by the co-founders and observers, as well as its activities from its inception to the present. Specifically, the jRCT*, a database of clinical trial information, has been identified as a problem by both those who search for information and those who input information, and the background for the establishment of the "Creation Society" was to urge the Ministry of Health, Labour and Welfare to upgrade the system to make it easier for all stakeholders to use. In December 2023, we submitted a written request to the MHLW for system modifications, and the January 2024 modifications have already been implemented, reflecting some of the requests submitted by the Creator's Group.
The MHLW will continue to hold meetings and make requests for system modifications in the future, and will also continue to consider activities to raise awareness and disseminate clinical trial information.
*Japan Registry of Clinical Trials

Closing Remarks

Mr. Yoshiyuki Ishida, Managing Director of the Japan Pharmaceutical Manufacturers Association (JPMA), expressed his gratitude to the participants, saying, "While there are many issues to be addressed to improve the healthcare environment, it is important for patient groups, medical professionals, and pharmaceutical companies to work together to solve each problem one by one. He also expressed his gratitude to the participants.

Free discussion

After the main program, a time was set aside for free discussion on the Web among the speakers, patient groups in the audience, and members of the Pharmaceutical Cooperative Drug Evaluation Committee. There was a lively exchange of opinions about what was not fully discussed and what they wanted to hear more about.

(Yasuhiro Marumoto, Leader of Mutual Understanding TF, Patient Group Collaboration Promotion Committee)

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